Baby's life, death a stem-cell drama

SACRAMENTO - Little Sarah Mooreland died two years ago at just 9 months old, but her parents hope her influence will endure for generations.

Lonni and Jay Mooreland moved from Folsom, Calif., to Minneapolis to help support other parents and raise funds for research into the rare skin disease that killed their daughter.

On Thursday, the couple's dedication got a boost when the prestigious New England Journal of Medicine published the first detailed report on the experimental stem-cell treatment their daughter had started to undergo.

Researchers found that stem cells from transplanted bone marrow travel to the skin, where they help produce a key protein that's lacking in people with genetic diseases like Sarah's.

The procedure has been used on just 13 children so far. No one knows whether some youngsters who have improved just a little will plateau where they are, keep getting better, or eventually backslide and get worse.

Even so, the experiment has been watched by stem-cell researchers around the country because, until now, the strongest evidence for stem-cell cures has been restricted to bone and blood ailments.

"We're incredibly encouraged by the Minnesota trial," said Jan Nolta, director of the University of California-Davis Institute for Regenerative Cures. "It's one of the first real proofs of principles that adult stem cells can help tissues outside of the blood-forming system."

The lead author of the new study, Dr. John Wagner of the University of Minnesota in Minneapolis, said the Moorelands' move to his city, and their ongoing support, has been "extraordinary."

The first child Wagner treated with a bone-marrow transplant for epidermolysis bullosa, or EB, did well.

The next two died, including baby Sarah, who did not last through the perilous preparatory stage of destroying her own immune system so she could receive donor bone marrow.

After one moderate success and two deaths, "for me to do Patient 4 was exceedingly hard," Wagner recalled. "Lonni said, 'You have to keep going.' And Patient 4 turned out to be the best."

The fourth child to get the treatment now can slide down a slide and bounce on a trampoline, stunning achievements for a child with such a disabling disease, Wagner said.

Several different genetic mutations cause EB by leaving the body unable to produce key ingredients needed for skin integrity. Depending on what's missing, people can have a relatively mild disease or one so devastating that a child is likely to die in the first year of life.

Sarah had recessive dystrophic EB, which leaves the upper layer of skin unable to anchor to the layer beneath it. The skin sloughs away from the body, and linings of the throat and stomach blister and erode.

People with recessive dystrophic EB must live swathed in bandages. They have so many unhealed wounds that toes and fingers can fuse together in what doctors call mitten deformities. Digestive problems leave them malnourished. The constant breakdown and rebuilding of their skin makes them prey to skin cancer. Most die in their teens and 20s.

The Moorelands moved to Minnesota in hopes that their daughter's fate might lead to knowledge that could help others.

It is not clear whether the long-term benefits of undergoing such a dangerous treatment will outweigh the risks.